CRISPR on the Cusp: The Promise and the Pain Points

Hereditary angioedema patients treated with Intellia’s NTLA-2002—an in vivo CRISPR-Cas9 therapy— had an average 95% reduction in the sudden, debilitating bouts of swelling that are a hallmark of the disease, with some patients remaining attack-free for more than a year, according to updated Phase I data presented in June. But despite the potential of gene editing to ease or even cure genetic diseases, questions about the safety and efficacy of the techniques remain.